Clinical trials: Newborn Diseases
Dermatitis, Atopic
Drug Drug Interaction Study Evaluating the Effect of Fluvoxamine or Fluconazole on PK and Safety of PF-04965842.
A Phase 1, Open Label, Parallel-Cohort, Randomized, Fixed Sequence Study To Evaluate The Pharmacokinetic Drug Drug Interaction Between PF-04965842 and Fkuvoxamine (cohort 1) or Fluconazole (Cohort 2) in healthy subjects.
- Trial name
- NCT03634345
- Testing
- Period 1 - Day 1: PF-04965842 administered, Period 2: Cohort 1: Fluvoxamine & PF-04965842, Period 2: Cohort 2: Fluconazole & PF-04965842
- Where
- Belgium
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
Thyroid Eye Disease, Graves' Orbitopathy
Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis With Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study
The overall objective is to investigate the efficacy, tolerability, and safety of teprotumumab (a fully human monoclonal antibody [mAb] inhibitor of the insulin-like growth factor-1 receptor [IGF-1R]) administered once every 3…
- Trial name
- OPTIC (NCT03298867)
- Testing
- Teprotumumab
- Where
- 14 sites in United States, Germany and 1 more
- Volunteers
- 83 people
- Sponsor
- Amgen
Neonatal Respiratory Distress
Aerosolized Infasurf Treatment Protocol: Aerosolized Infasurf for Spontaneously Breathing Patients With Early RDS (AERO-03)
Surfactant replacement therapy can be life-saving for newborn infants born with symptomatic lung surfactant deficiency causing Respiratory Distress Syndrome (RDS).
- Trial name
- NCT03582930
- Testing
- Infasurf Aero
- Where
- 5 sites in United States
- Sponsor
- ONY
Cystic Fibrosis Lung
4D-710 in Adult Patients With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Trial name
- CF (NCT05248230)
- Testing
- 4D-710
- Where
- 20 sites in United States
- Volunteers
- 30 people
- Sponsor
- 4D Molecular Therapeutics
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
A Study on Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in Children, Teenagers and Adults in Portugal
The main aim of this study is to learn about the percentage of persons diagnosed with Dravet Syndrome (DS) and Lennox-Gastaut-Syndrome (LGS) in 2022 and of persons newly diagnosed in 2021 and 2022 compared to the overall…
- Trial name
- DRALEGA-PT (NCT06395792)
- Testing
- No Intervention
- Sponsor
- Takeda
Erythropoietic Protoporphyria
Phase III Confirmatory Study in Erythropoietic Protoporphyria
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Between 75 and 100 eligible patients will be enrolled.
- Trial name
- NCT01605136
- Testing
- Afamelanotide
- Where
- 7 sites in United States
- Volunteers
- 93 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Thyroid Eye Disease
An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with active Thyroid Eye Disease (TED).
- Trial name
- REVEAL-1 (NCT06625411)
- Testing
- VRDN-003
- Where
- 48 sites in United States, Germany and 3 more
- Volunteers
- 132 people
- Sponsor
- Viridian Therapeutics, Inc.
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
Hemophilia A, Hemophilia B
Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors.
- Trial name
- MATCHBOX (NCT01921855)
- Testing
- BAY Factor VII (BAY86-6150)
- Where
- 4 sites in Poland, South Africa and 1 more
- Volunteers
- 16 people
- Sponsor
- Bayer
Hydrocephalus, Nervous System Diseases
Study of Shunt Flow Sensor Accuracy in Extra-ventricular Drains.
The study hypothesis is that an ultrasonic flow sensor can accurately measure flow in hydrocephalic shunts. The ultrasonic sensor will measure cerebrospinal fluid drainage in hydrocephalus patients with external ventriculostomies…
- Conditions
- Hydrocephalus, Nervous System Diseases, Infant, Newborn Diseases
- Trial name
- NCT01108965
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Transonic Systems Inc.
Cystic Fibrosis
Study of Denufosol Inhalation Solution in Patients With Mild to Moderate Cystic Fibrosis Lung Disease
The purpose of this trial is to evaluate the effects of a single dose of denufosol versus placebo on mucociliary clearance in patients with mild to moderate CF lung disease
- Trial name
- NCT00425165
- Testing
- denufosol tetrasodium (INS37217) Inhalation Solution
- Volunteers
- 6 people
- Sponsor
- Merck Sharp & Dohme LLC
ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant…
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Trial name
- NCT05030831
- Testing
- INZ-701
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Inozyme Pharma
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Rheumatoid Arthritis, Osteoarthritis
A Single Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Ceramic-on-ceramic Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Pinnacle™ Cup with a ceramic-on-ceramic bearing combination in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872222
- Testing
- Pinnacle™ Acetabular System
- Where
- Czechia
- Volunteers
- 98 people
- Sponsor
- DePuy International
Atopic Dermatitis
Safety, Tolerance and Pharmacokinetics Clinical Study of VC005 in Healthy Subjects and Patients with Mild to Moderate Atopic Dermatitis
This study is a single center, randomized, double-blind, Vehicle controlled,, single and multiple dose clinical study.
- Trial name
- NCT06009094
- Testing
- VC005
- Where
- China
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
Electroencephalographic Neonatal Seizures
A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures
The purpose of the study is to evaluate the pharmacokinetics (PK) of brivaracetam (BRV) in neonates who have seizures that are not adequately controlled with previous antiepileptic drug (AED) treatment, and to identify the…
- Trial name
- NCT03325439
- Testing
- Brivaracetam (BRV) intravenous (iv), Brivaracetam (BRV) oral
- Where
- 12 sites in Belgium, Czechia and 5 more
- Volunteers
- 9 people
- Sponsor
- UCB Biopharma SRL
Alpha 1-Antitrypsin Deficiency, Emphysema
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
- Trial name
- ELEVAATE (NCT05856331)
- Testing
- SAR447537, Zemaira
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 99 people
- Sponsor
- Sanofi
Hypophosphatasia
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.
- Trial name
- CHESTNUT (NCT06079372)
- Testing
- ALXN1850, asfotase alfa
- Where
- 21 sites in United States, Argentina and 5 more
- Volunteers
- 43 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Spina Bifida
Establishing the Preliminary Utility of a Novel Pediatric Manual Mobile Standing Wheelchair
To establish the preliminary utility of a novel pediatric manual mobile standing wheelchair (PedMMSWC).
- Trial name
- NCT07290556
- Testing
- PedMMSWC Wheelchair User Testing, PedMMSWC Simulated Home, School, Community Environment
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Optimal Mobility, Inc
Complication of Renal Dialysis, End Stage Renal Disease
A Study Testing the Use of a Perivascular Sirolimus Formulation (Sirogen) in ESRD Patients Undergoing AV Fistula Surgery
The primary study objective is to evaluate the benefit of the Sirolimus eluting Collagen implant (SeCI; Sirogen), a single dose prophylactic treatment delivered intraoperatively at the time of surgical creation of an…
- Conditions
- Complication of Renal Dialysis, End Stage Renal Disease, End Stage Kidney Disease, ESRD
- Trial name
- ACCESS2 (NCT05425056)
- Testing
- Sirolimus
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 136 people
- Sponsor
- Vascular Therapies, Inc.
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